LNP.UCD.ABE

LNP.UCD.ABE is an investigational lipid nanoparticle (LNP)-encapsulated adenine base editor (ABE) therapy designed for the treatment of various urea cycle disorders (UCDs). Developed by researchers at the Children's Hospital of Philadelphia, including Rebecca Ahrens-Nicklas, the therapy utilizes a base editing approach to precisely correct pathogenic variants in genes involved in the urea cycle, such as CPS1, OTC, ASS1, ASL, ARG1, NAGS, and SLC25A15. By delivering the ABE machinery intravenously via LNPs, the treatment aims to restore functional enzyme activity in the liver, potentially providing a durable cure for pediatric patients with severe infantile-onset UCDs. It is currently being evaluated in a Phase 1/2 umbrella clinical trial.